Status: Finalised First registered on: 30/01/2017
Last updated on: 30/01/2017
1. Study identification
EU PAS Register NumberEUPAS17462
Official titleOUTCOMES OF THE SPANISH COHORT OF EARLY ACCESS TO PERTUZUMAB AND TRASTUZUMAB EMTANSINE
Study title acronymKNOWHER STUDY
Study typeObservational study
Brief description of the studyCurrently, four targeted anti-HER2 agents are available in the EU for the treatment of advanced HER2+ breast cancer: trastuzumab (a humanized monoclonal antibody that targets subdomain IV of HER2), lapatinib (a HER1/HER2 dual tyrosine kinase inhibitor) and, more recently, pertuzumab (a humanized monoclonal antibody that targets domain II of HER2) and trastuzumab emtansine (an antibody-drug conjugate). Three of these agents have been approved in combination with chemotherapy: trastuzumab, lapatinib (also authorized in combination with trastuzumab), and pertuzumab. Trastuzumab emtansine (T-DM1) is the only targeted agent currently approved as monotherapy. The addition of pertuzumab (Perjeta®) to trastuzumab plus chemotherapy (docetaxel) in first line treatment of HER2-positive metastatic breast cancer obtains a significant and clinically relevant increase in median PFS of 6.1 months. Both Kadcyla® and Perjeta® were available under those special access systems. In order to be eligible for CU of Trastuzumab emtansine (T-DM1) or Pertuzumab, patients had to meet the some prespecified criteria. After the EU approval, oncologists could prescribe the product to specific patients through the Early Access Program, provided that the local or regional responsible people accept payment. In order to evaluate the effectiveness and safety of Trastuzumab emtansine (T-DM1) and Pertuzumab in HER2-positive metastatic breast cancer under real-world disease conditions, is proposed a retrospective observational cohort non-comparative study / registry in Spain.
Was this study requested by a regulator?No
Is the study required by a Risk Management Plan (RMP)? Not applicable
Regulatory procedure number (RMP Category 1 and 2 studies only)
Other study registration identification numbers and URLs as applicableClinicalTrials.gov Identifier: NCT03025711 https://clinicaltrials.gov/ct2/show/NCT03025711?term=NCT03025711&rank=1
2. Research centres and Investigator details
Coordinating study entity
Centre nameHospital Universitario Puerta de Hierro
Centre locationMadrid. Spain
Details of (Primary) lead investigator
Title Dr
Last name Cristina
First name Avendaño-Sola
Is this study being carried out with the collaboration of a research network?
No
Other centres where this study is being conducted
Multiple centres

In total how many centres are involved in this Study?70

Clinical Pharmacology Service HUPHM
Hospital Clinic Barcelona
Countries in which this study is being conducted
National study

Spain
3. Study timelines: initial administrative steps, progress reports and final report
PlannedActual
Date when funding contract was signed01/12/201601/12/2016
Start date of data collection03/04/201730/01/2017
Start date of data analysis03/07/201730/01/2017
Date of interim report, if expected
Date of final study report31/10/201730/01/2017
4. Sources of funding
Please provide estimates of the percentage of funding by source for this study
Names(s)Approximate % funding
Pharmaceutical companiesRoche Farma, S.A100
Charities
Government body
Research councils
EU funding scheme
5. Contact details for enquiries
Scientific Enquiries
Title Dr
Last name Cristina
First name Avendaño-Sola
Address line 1Clinical Pharmacology Service
Address line 2Manuel de Falla 1 
Address line 3 
CityMajadahonda 
Postcode282222 
CountrySpain
Phone number (incl. country code)34911917479 
Alternative phone number 
Fax number (incl. country code) 
Email address mariabelen.ruiz@salud.madrid.org
Public Enquiries
Title Dr 
Last name Cristina 
First name Avendaño-Sola 
Address line 1Clinical Pharmacology Service 
Address line 2Manuel de Falla 1 
Address line 3 
CityMajadahonda 
Postcode282222 
CountrySpain 
Phone number (incl. country code)34911917479 
Alternative phone number 
Fax number (incl. country code) 
Email address mariabelen.ruiz@salud.madrid.org 
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